Fig. 4
From: Gene therapy for ultrarare diseases: a geneticist’s perspective
The mechanism of AAV vector delivery to the brain. AAV vectors can be administered directly into the brain parenchyma or injected into CSF spaces (e.g., by lumbar puncture) and the vectors then migrate to the spinal cord and brain. AAVs can transduce neural cells in the brain and ameliorate their dysfunction. Transgenes may be secreted to alleviate the dysfunction of other cells